Human, mouse PD-1 differ inside and out
The PD-1 receptor, a major immune checkpoint inhibitor whose signaling is the target of multiple blockbuster anticancer drugs, differs functionally between rodents and humans in previously unknown ways. Researchers from the University of California, San Diego, and co-authors at the Chinese Academy of Sciences and the National Cancer Institute reported these findings in the Jan. 3, 2025, online issue of Science Immunology.

Human, mouse PD-1 differ inside and out
The PD-1 receptor, a major immune checkpoint inhibitor whose signaling is the target of multiple blockbuster anticancer drugs, differs functionally between rodents and humans in previously unknown ways. Researchers from the University of California, San Diego, and co-authors at the Chinese Academy of Sciences and the National Cancer Institute reported these findings in the Jan. 3, 2025, online issue of Science Immunology.
AD-353 and AD-408, S1R antagonists with efficacy in models of mechanical hyperalgesia and allodynia
Researchers from Vera Salus Ricerca Srl and affiliated organizations have reported the discovery and preclinical characterization of novel sigma-1 receptor (S1R) antagonists as potential new analgesic agents.
Affinia names development candidate for BAG3 dilated cardiomyopathy
Affinia Therapeutics Inc. has nominated AFTX-201 as a development candidate for the treatment of BAG3 dilated cardiomyopathy. The gene therapy, using Affinia’s cardiotropic capsid, is given as a one-time intravenous injection.
Frontier Medicines selects covalent p53 Y220C activator
Frontier Medicines Corp. has announced the selection of FMC-220, a first-in-class covalent small-molecule activator of p53 Y220C designed to address potency and tolerability challenges of noncovalent approaches. In cancer with mutations, the Y220C mutation destabilizes the p53 protein, impairing its tumor-suppressive functions and contributing to cancer progression.
Mural Oncology nominates candidates from IL-18 and IL-12 programs
Mural Oncology plc has nominated two new development candidates, one for its interleukin-18 (IL-18) program and one for its IL-12 program.
2024 BioWorld Science Year in Review
Taking a look back at the achievements in research this year, including brain maps, pandemic breakthroughs and advances in pregnancy health.
2024 BioWorld Year in Review
BioWorld’s staff recaps the therapeutic trends and breakthroughs of 2024, the economic highs and lows facing the industry throughout the past year and the regulatory actions that are likely to have implications as biopharma forges ahead in 2025.
2024 BioWorld MedTech Year in Review
BioWorld MedTech’s staff recaps the med-tech and diagnostic trends and breakthroughs of 2024, the economic highs and lows facing the industry throughout the past year and the regulatory actions that are likely to have implications as med tech forges ahead in 2025.
Potent, selective MTHFD2 inhibitor shows favorable PK and antitumor efficacy in AML models
Scientists from Taiwan have detailed the discovery and characterization of new MTHFD2 inhibitors.
Shanghai Yingli Pharmaceutical patents new PARG inhibitors
Shanghai Yingli Pharmaceutical Co. Ltd. has disclosed new compounds.
Series A financing at Rheumagen supports HLA gene-editing therapies for autoimmune diseases
The financing will enable progression of RG-0401.

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Conferences

By targeting HPA-1a, RLYB-212 prevents rare fetal blood disorder
American Society of Hematology
Fetal/neonatal Alloimmune Thrombocytopenia (FNAIT) is a bleeding disorder caused by maternal alloantibodies against fetal platelets during pregnancy.
CCRL2 is therapeutic target in acute erythroid leukemia
American Society of Hematology
C-C chemokine receptor-like 2 (CCRL2) is a chemokine receptor involved in inflammation activation and is usually expressed in differentiated myeloid cells. CCRL2 is overexpressed in acute erythroid leukemia (AEL) cells compared to healthy cells. Antibody-drug conjugates (ADCs) against CCRL2 in AEL...
Seismic’s S-1117 reduces IgG levels in autoantibody-driven disorders
American Society of Hematology
The effects of S-1117 regarding IgG cleavage and degradation were tested in vitro.
New MALT1 degrader shows potent activity in preclinical lymphoma models
New compound
The protease mucosa-associated lymphoid tissue lymphoma translocation 1 (MALT1) is a signaling protein with both molecular scaffolding and protease activity involved in lymphocyte activation. MALT1 is considered a therapeutic target for chronic lymphocytic leukemia (CLL) in patients who develop...

Cancer

SKA1 gene linked to cancer progression and radioresistance in oral squamous cell carcinoma study
Oral squamous cell carcinoma (OSCC), a major subset of head and neck cancers, is known for its high mortality rates and limited treatment options.

Infection

Zydus Lifesciences discovers new compounds to treat dengue virus infection
Patents
Zydus Lifesciences Ltd. has described compounds reported to be useful for the treatment of dengue virus infections.

New selective and potent ClpP agonist effectively targets S. aureus, including MRSA
Caseinolytic protease P (ClpP) is a conserved serine protease present in both bacteria and humans, essential for maintaining protein quality by degrading misfolded proteins.
In vivo morpholino oligomers targeting the 3′ untranslated region show efficacy against dengue virus
The dengue virus (DENV) is a single-stranded RNA virus with increasing worldwide prevalence causing severe mosquito-borne viral infections. Therefore, effective strategies to prevent and treat DENV infections are urgently needed.

Neurology/psychiatric

Tenvie Therapeutics launches with focus on neurological diseases
Drug design, drug delivery and technologies
The company is advancing a pipeline of therapeutics, including programs acquired from Denali Therapeutics Inc., focused on treating neurological, cardiometabolic and ophthalmic diseases.
Series A financing at Alesta Therapeutics to advance programs in hypophosphatasia and CMT
Financings
Alesta Therapeutics BV has closed an oversubscribed €65 million (US$67 million) series A financing round designed to support development of its two small-molecule therapeutics.
Map is first step toward healthy brains into old age
Science
2024 saw the completion of several cellular-resolution brain maps, including the entire fly brain and a comprehensive connections map of a cubic centimeter of human brain. 2025 began with the addition of another important map. In the Jan. 1, 2025, issue of Nature, researchers from the Allen...
Australia’s Celosia heads toward clinic with gene therapy for ALS
Financings
After raising AU$16.75 million (US$10.4 million) in a series A round, Celosia Therapeutics Pty Ltd. is heading toward the clinic with its novel gene therapy that targets TDP-43, a protein directly linked to amyotrophic lateral sclerosis (ALS) pathology.
Novel NLRP3 inhibitor shows neuroprotective effect
Neuroinflammation is a key feature of Alzheimer’s disease (AD). The activation of the NLRP3 inflammasome has been associated with the pathogenesis of AD, therefore, targeting NLRP3 may help reduce neuroinflammation and slow down the progression of AD.

Endocrine/metabolic

Dual and triple agonists of GCG, GIP and GLP-1 receptors disclosed in Sun Pharmaceutical patent
Patents
Sun Pharmaceutical Industries Ltd. has divulged incretin analogues acting as dual agonists of gastric inhibitory polypeptide (GIPR) receptor/GLP-1 or GLP-1/glucagon receptor (GCGR) or triple agonists of GIPR/GLP-1/GCGR reported to be useful for the treatment of type 2 diabetes, dyslipidemia,...

Biomarkers

Circulating splicing factor SRRM1: a potential biomarker and therapeutic target in prostate cancer
Cancer
Prostatic-specific antigen (PSA) is commonly used as a screening tool for prostate cancer but presents limited sensitivity and specificity. Therefore, research efforts are focused on searching for novel noninvasive diagnostic biomarkers.

miR-1-3p levels predict case severity in hospitalized COVID-19 patients
Infection
Increasing evidence shows that circulating microRNAs (miRNAs) regulate biological processes involved in COVID-19 complications.
Biomarker could aid in psychosis diagnosis
Science
Researchers at the University of Rochester have described a neuroimaging-based biomarker that could identify individuals with early psychosis, and improved their identification when it was added to a standard neurocognitive diagnostic test. In a group of roughly 160 participants in the Human...

Gastrointestinal

Flagship Pioneering Innovations VI divulges new CD38 inhibitors
Patents
Flagship Pioneering Innovations VI LLC has synthesized ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 (CD38) inhibitors.
KHK inhibitors disclosed in Centennial Therapeutics patent
Patents
Centennial Therapeutics LLC has divulged ketohexokinase (KHK) inhibitors reported to be useful for the treatment of nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH).
NIDDK grant supports Orphagen’s RARα antagonist for inflammatory bowel disease
Grant
Orphagen Pharmaceuticals Inc. has been granted an award of up to $1.7 million.
Amgen discloses 15-PGDH inhibitors
Patents
Amgen Inc. has reported new 5,6- and 6,6-fused bicyclic alcohols and ethers acting as 15-hydroxyprostaglandin dehydrogenase (15-PGDH) inhibitors. They are reported to be useful for the treatment of gastrointestinal disorders.
SRT-015 prevents acute liver injury in preclinical tests
Conferences
Genfit SA and its collaborators have described the preclinical efficacy of SRT-015, a liver-selective ASK1 inhibitor in models of liver failure.
EA-3571, a dual inhibitor of enteropeptidase and trypsin with efficacy in mouse model of MASH
Conferences
Recently presented preclinical data show that EA Pharma Co. Ltd.'s EA-3571 is a highly potent dual inhibitor of enteropeptidase and trypsin with luminal action, resulting in potential anti-insulin resistance and fat-burning properties.

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